Listing of Pharmaceutical Benefits (NHL) - Schedule 4 part 1

Familial homozygous hypercholesterolaemia

Initial treatment

The treatment must be in conjunction with dietary therapy and exercise; AND

The condition must have been confirmed by genetic testing; or

The condition must have been confirmed by a Dutch Lipid Clinic Network Score of at least 7; AND

Patient must have an LDL cholesterol level in excess of 1.8 millimoles per litre; AND

Patient must have been treated with the maximum recommended dose of atorvastatin (80 mg daily) or rosuvastatin (40 mg daily) according to the TGA-approved Product Information or the maximum tolerated dose of atorvastatin or rosuvastatin for at least 12 consecutive weeks in conjunction with dietary therapy and exercise; or

Patient must have developed clinically important product-related adverse events necessitating withdrawal of statin treatment to trials of each of atorvastatin and rosuvastatin; or

Patient must be contraindicated to treatment with a HMG CoA reductase inhibitor (statin) as defined in the TGA-approved Product Information; AND

Must be treated by a specialist physician; or

Must be treated by an authorised prescriber in consultation with a specialist physician.

The qualifying LDL cholesterol level following at least 12 consecutive weeks of treatment with a statin (unless treatment with a statin is contraindicated or following completion of statin trials as described in these prescriber instructions in the event of clinically important adverse events) must be documented in the patient's medical records and must be no more than 8 weeks old.

A clinically important product-related adverse event is defined as follows:

(i) Severe myalgia (muscle symptoms without creatine kinase elevation) which is proven to be temporally associated with statin treatment; or

(ii) Myositis (clinically important creatine kinase elevation, with or without muscle symptoms) demonstrated by results twice the upper limit of normal on a single reading or a rising pattern on consecutive measurements and which is unexplained by other causes; or

(iii) Unexplained, persistent elevations of serum transaminases (greater than 3 times the upper limit of normal) during treatment with a statin.

The following must be documented in the patient's medical records:

(i) the qualifying Dutch Lipid Clinic Network Score; or

(ii) the result of genetic testing confirming a diagnosis of familial homozygous hypercholesterolaemia

One of the following must be documented in the patient's medical records regarding prior statin treatment:

(i) the patient was treated with atorvastatin 80 mg or rosuvastatin 40 mg or the maximum tolerated dose of either for 12 consecutive weeks; or

(ii) the doses, duration of treatment and details of adverse events experienced with trials with each of atorvastatin and rosuvastatin; or

(iii) the patient is contraindicated to treatment with a statin as defined in the TGA-approved Product Information.

Patients with symptomatic atherosclerotic cardiovascular disease where LDL cholesterol cannot be measured due to hypertriglyceridaemia, may qualify under this authority application if they have a non-HDL in excess of 2.4 millimoles per litre.